Leading RNAi therapeutics company, Alnylam Pharmaceuticals, Inc. has formed a collaboration with a leading vaccine manufacturer, GlaxoSmithKline (GSK) focused on Alnylam's VaxiRN technology for certain GSK vaccine products, including influenza. GSK is the first company to form a collaboration accessing this new platform. VaxiRNA is a new RNAi technology for the enhanced production of viruses used in the manufacture of vaccine products.

Alnylam is applying RNAi technology to improve the manufacturing processes for vaccines in an effort called VaxiRNA. With VaxiRNA, Alnylam is using siRNAs that silence specific genes in vaccine production systems, such as cells or eggs, which limit or prevent the efficient growth of viruses used in the manufacture of vaccine products. New innovations in vaccine manufacturing are needed to enable the scale and speed of global immunization for a number of pathogens. In 2011, Alnylam formed a VaxiRNA partnership with GlaxoSmithKline for influenza vaccine production.

John Maraganore, chief executive officer, Alnylam said, "At Alnylam, we continue to pioneer innovative opportunities for our RNAi platform that could have transformative impact in medicine and the biopharmaceutical industry. This is exemplified with VaxiRNA, where siRNA technology is being used to increase virus titers in the production of vaccine products. Since production of vaccines can be limiting for the scale and speed of global immunization against many pathogens, including influenza, we believe that VaxiRNA can become an enabling technology for the broader vaccine industry."

 "As we advance our VaxiRNA effort, we are excited to form a new collaboration with GSK, a global leader and innovator in vaccine development and manufacturing. With GSK, we aim to apply VaxiRNA technology to enhance influenza vaccine production in cell culture, where Alnylam will receive research funding, in addition to potential milestones and payments on unit sales of commercialized vaccine product, " John added.

Alnylam's VaxiRNA platform applies siRNAs for the silencing of specific genes that limit or prevent efficient growth of viruses in vaccine manufacturing systems, including those in cell culture and also in eggs. The VaxiRNA platform stems from work Alnylam has performed as part of its Alnylam Biotherapeutics efforts, where RNAi technology has been applied to improve the manufacture of biotherapeutic products, such as recombinant proteins and monoclonal antibodies. This new platform addresses the significant unmet commercial need for innovative technologies that can improve the manufacture of vaccine products, especially where vaccine production is a limiting factor for the scale and speed of global immunization needs.

The GSK collaboration is focused initially on influenza vaccine production in cell culture systems. Under the terms of the agreement, GSK will provide funding and certain milestone payments to Alnylam. If successfully applied in the manufacture of commercial product, Alnylam will receive payments on unit product sales. In addition, GSK has obtained an option for VaxiRNA applications toward two additional vaccine products.

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia.